Tiziana seeking FDA OK to launch clinical trial of foralumab in ALS
Phase 2 trial to test safety, early efficacy of nasal spray in 20 patients
Tiziana Life Sciences is seeking regulatory clearance in the U.S. to launch a Phase 2 clinical trial evaluating foralumab nasal spray, its lead therapeutic candidate, as a treatment for amyotrophic lateral sclerosis (ALS).
The filing of the company’s investigational new drug application with the U.S. Food and Drug Administration (FDA) comes on the heels of a key grant awarded by the ALS Association to support an early-stage trial of intranasal foralumab.
Pending a green light from the FDA, the study will test two doses of the experimental medication, given through a spray into the nose, in 20 ALS patients. The goal is to assess the treatment’s safety and early signs of efficacy.
“This filing represents a significant achievement for our team and underscores our commitment to addressing the urgent medical needs of ALS patients,” Ivor Elrifi, Tiziana’s CEO, said in a company press release.
ALS Association award is supporting clinical trial of foralumab
Tiziana’s foralumab is an antibody-based therapy that has demonstrated its ability to reduce harmful brain inflammation in neurodegenerative diseases such as ALS, multiple sclerosis (MS), and Alzheimer’s disease.
By binding to CD3, a protein present on the surface of T-cells, foralumab reduces the activity of inflammatory T-cells while boosting the function of regulatory T-cells — a specialized immune subset that suppresses the inflammatory activity of other immune cells.
This dual mechanism allows foralumab to effectively modulate T-cell function and reduce harmful responses from other immune cells involved in brain inflammation, including the brain-resident immune cells microglia. This may help to prevent nerve cell damage and slow disease progression.
We are excited to initiate this important clinical study with the support of the ALS Association grant.
In an Alzheimer’s mouse model, intranasal foralumab reduced microglial activity to normal levels and improved cognitive function. These effects were associated with regulatory T-cells traveling into the brain and interacting with microglia.
In MS, foralumab has shown encouraging results in clinical settings. Under an expanded patient access program — which enables patients with serious or life-threatening conditions to access experimental therapies outside clinical trials — the treatment reduced microglial activity and improved fatigue levels in most patients using the nasal spray for at least six months.
Encouraged by its success in reducing microglial activation in Alzheimer’s and MS, Tiziana is now advancing its foralumab nasal spray into an early-stage trial for ALS.
No further details about the trial were shared at this point, but the company had previously announced that it would last six months and would use advanced imaging techniques, including PET scans, to measure changes in microglia activity and other markers of brain inflammation.
The funding from the ALS Association was via its Hoffman ALS Clinical Trial Awards Program, which is designed to support early clinical studies testing new therapies. The program’s goal is to try to speed the development of new treatments and help garner additional funding.
“We are excited to initiate this important clinical study with the support of the ALS Association grant,” Elrifi said.
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